What Graphite Bio does

Graphite Bio was a clinical-stage biotechnology company developing CRISPR/Cas9-based ex vivo gene-edited cell therapies, with its lead program GPH101 aimed at sickle cell disease (SCD) by correcting the disease-causing HBB gene mutation in autologous hematopoietic stem cells. Founded in 2020 and headquartered in South San Francisco, the company was built on foundational research from co-founders including Stanford's Dr. Matthew Porteus, a recognized leader in therapeutic genome editing. The business model was a pre-revenue, capital-intensive biotech playbook: raise venture and public equity, advance a single asset through early clinical development, and target eventual partnership, acquisition, or commercialization with a larger biopharma player.

Operationally, the company concentrated its resources on GPH101, which entered a Phase 1/2 CEDAR trial in SCD. The company went public on NASDAQ in mid-2021 (ticker GRPH) and raised approximately $150.7 million in a financing round shortly thereafter, on top of a $45 million Series A in September 2020. In early 2023, Graphite Bio voluntarily paused the CEDAR trial following a serious adverse event (prolonged cytopenia) in a treated patient, materially damaging the program's risk profile. Subsequently, the company delisted from NASDAQ and its corporate web presence appears to have been absorbed by or transferred to LENZ Therapeutics, an unrelated ophthalmology-focused biotech, indicating effective cessation of independent operations as a gene editing entity.

Name

Graphite Bio

Legal name

Graphite Bio

Website

https://graphitebio.com

Company type

Private

Founded year

2020

Operating status

Operating

Headcount range

51–100 employees

Short description

Graphite Bio was a pre-revenue, clinical-stage gene editing company developing CRISPR-based ex vivo cell therapies, with lead candidate GPH101 aimed at correcting the sickle cell disease mutation in autologous hematopoietic stem cells.

Ownership category

akta.pro rank

Product category

Gene Therapy Biopharmaceuticals

NAICS

Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Stem Cell Therapies (MSC/iPSC/HSC-derived) (HLAAABAE)

akta.pro secondary industries

Stem Cell–Based Regenerative Therapies (HLAAAGAA), Gene Therapy (Viral Vector/Plasmid) CDMO (HLAGABAE)

Keywords

• Gene editing therapy
• Cell therapy development
• CRISPR biotechnology
• Sickle cell treatment
• Genetic disease therapeutics

Headquarters

HQ city

South San Francisco

HQ country

United States

HQ region

North America

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Operations, Infrastructure

Core offering

Graphite Bio is a biotechnology company developing ex vivo gene-edited cell therapies, with its lead product candidate GPH101 designed to correct the HBB gene mutation in patient-derived stem cells and restore normal adult hemoglobin production for the treatment of sickle cell disease. The therapy involves extracting a patient's hematopoietic stem cells, editing them using a CRISPR-based platform, and reinfusing the corrected cells. GPH101's clinical development was voluntarily paused in early 2023 after patients experienced adverse side effects.

Product overview

Graphite Bio is a biotechnology company developing gene-editing therapies. The company's primary product candidate is GPH101, an ex vivo gene-edited cell therapy for sickle cell disease that aims to correct the HBB gene mutation to restore normal adult hemoglobin production.

Differentiator

Problem solved

Functional benefit

Products and services

• GPH101 An ex vivo gene-edited cell therapy candidate for sickle cell disease designed to correct the HBB gene mutation in patient-derived hematopoietic stem cells to restore normal adult hemoglobin production. Development was halted following voluntary trial pause in early 2023 due to adverse patient events.

Quantifiable outcome

• Clinical trials for GPH101 were voluntarily paused in early 2023 after patients developed adverse side effects, halting development progress

Segments1 record

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Recent moves6 records

Expansion highlights3 records

Direct peers

• CRISPR Therapeutics: Co-developer of Casgevy (exagamglogene autotemcel) with Vertex, the first FDA-approved CRISPR-based ex vivo gene-edited cell therapy for sickle cell disease. Most direct competitor to Graphite Bio's GPH101, using a BCL11A erythroid-enhancer disruption approach in patient HSCs.
• Editas Medicine: Developed EDIT-301 (reni-cel), an ex vivo CRISPR-edited HSC therapy for sickle cell disease and beta-thalassemia. Direct competitor to Graphite Bio in autologous HSC gene editing for hemoglobinopathies, also paused/wound down in 2023 amid safety and strategic challenges.
• Sangamo Therapeutics: Pioneer in genome editing using zinc finger nucleases (ZFN), with multiple ex vivo autologous cell therapy programs for hemoglobinopathies including BIVV003 for sickle cell disease. Direct peer in autologous HSC gene editing for sickle cell.

Broad incumbents

• Vertex Pharmaceuticals: Commercialization partner for Casgevy and a dominant rare-disease biotech with established commercial infrastructure for gene and cell therapies. Represents both the dominant competitive force and a potential strategic acquirer for assets like GPH101.

Emerging players

• Beam Therapeutics: Pioneering base-editing platform developing HSC-targeted therapies for sickle cell disease (BEAM-101) using a different precision gene-editing modality. Comparable in target indication, modality (autologous HSC editing), and clinical stage, with a differentiated chemistry approach.
• Intellia Therapeutics: Clinical-stage CRISPR/Cas9 company developing both in vivo and ex vivo gene-editing therapies across multiple indications. Comparable as a peer gene-editing platform with overlapping scientific founders and investors, though currently focused on in vivo applications.
• Prime Medicine: Developer of prime editing, a next-generation precision gene-editing modality applicable to sickle cell disease (potential ex vivo HSC correction). Comparable as a clinical-stage gene-editing peer with similar therapeutic ambitions and modality profile.
• Verve Therapeutics: Clinical-stage precision gene-editing company focused on cardiovascular disease using both base editing and in vivo CRISPR approaches. Comparable as a next-generation gene-editing platform developer with shared scientific approach and investor base.
• Caribou Biosciences: Clinical-stage allogeneic CAR-T and gene-editing platform using CRISPR-based chRDNA technology. Comparable as a CRISPR-platform peer with cell therapy applications, though focused on oncology rather than hemoglobinopathies.
• Cellectis: Clinical-stage gene-editing and CAR-T company using TALEN-based gene editing for both autologous and allogeneic cell therapies. Comparable as a peer in ex vivo gene-edited cell therapy development, with overlapping platform technology.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat2 records

Key risks6 records

Key highlights5 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles9 records

Funding overview

Funding rounds2 records

Investors14 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Graphite Bio